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"The CHERISH study, conducted in collaboration with Ionis, further demonstrates the meaningful impact SPINRAZA can have in children with later-onset SMA, and reaffirms the benefit of treatment across SMA populations"

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Biogen
(NASDAQ:BIIB) will present Phase 3 end of study SPINRAZA® (nusinersen)
data from CHERISH, which demonstrated a highly statistically significant
and clinically meaningful improvement in motor function in children with
later-onset (most likely to develop Type 2 or Type 3) spinal muscular
atrophy (SMA) compared to untreated children. The overall findings
continue to support the robust efficacy and favorable safety profile of
SPINRAZA across a broad range of individuals with SMA. The SPINRAZA
development program represents the largest body of clinical data of its
kind in SMA. SPINRAZA data will be presented at the American Academy of
Neurology (AAN) annual meeting in Boston, Mass., April 22-28, 2017.

“The CHERISH study, conducted in collaboration with Ionis, further
demonstrates the meaningful impact SPINRAZA can have in children with
later-onset SMA, and reaffirms the benefit of treatment across SMA
populations,” said Alfred Sandrock, M.D., Ph.D., executive vice
president and chief medical officer at Biogen. “Our clinical development
program demonstrates the impact of early treatment, which is confirmed
by NURTURE data showing significant motor milestone improvements
generally consistent with normal development in presymptomatic infants
treated with SPINRAZA.”

CHERISH: Later-onset SMA (Most Likely to Develop Type 2 or Type 3)

CHERISH is a Phase 3, multicenter, randomized, double-blind,
sham-procedure controlled study to assess the efficacy and safety of
SPINRAZA in children with later-onset SMA. The 15-month study
investigated SPINRAZA in 126 non-ambulatory children 2 to 12 years old
who experienced symptom onset at greater than 6 months of age.

In the CHERISH end of study analysis, children on SPINRAZA demonstrated
a highly statistically significant and clinically meaningful improvement
in motor function, as observed by the treatment difference of 4.9 points
in the mean change from baseline to Month 15 in the Hammersmith
Functional Motor Scale Expanded (HFMSE) score (p=0.0000001). The HFMSE
is a validated tool specifically designed to assess motor function in
children with SMA. When measuring changes from baseline, children who
received SPINRAZA (n=84) achieved a 3.9 point mean improvement at Month
15, while children who were not on treatment (n=42) experienced a mean
decline of 1.0 point. Primary endpoint results of the end of study
analysis were consistent with results observed at the interim analysis.

Data from the other endpoints analyzed, including attainment of new
motor milestones and upper limb motor function, were consistently in
favor of children who received treatment.

SPINRAZA demonstrated a favorable safety profile. Treatment-emergent
adverse events (AEs), severe AEs and serious AEs (SAEs) were reported
less frequently in children treated with SPINRAZA than those not on
treatment. The majority of the AEs were considered to be either related
to SMA disease, common events in the general population, or events
related to the lumbar puncture procedure. No children discontinued the
study due to AEs.

“In CHERISH, most children with later-onset SMA treated with SPINRAZA
saw improvements in motor function and stabilization or slowing of
disease progression,” said Dr. Richard Finkel, chief of neurology,
Nemours Children's Hospital, Orlando, Florida. “As a physician who has
spent 37 years treating children with SMA, it’s incredibly encouraging
to see some patients on SPINRAZA achieve milestones such as crawling and
standing with assistance within the clinical trial. These kinds of
clinically meaningful improvements are unprecedented and give new hope
to individuals with SMA and their families.”

NURTURE: Presymptomatic Infants with SMA

Biogen will also present new interim data from the Phase 2, multicenter,
open-label, single-arm NURTURE study evaluating SPINRAZA for the
treatment of infants under six weeks old with genetically diagnosed SMA
who were presymptomatic at treatment initiation. At the time of the
interim analysis, infants (n=20) were enrolled for a median of 317.5
days, and all infants were alive and none required respiratory
intervention (chronic non-invasive ventilation, invasive ventilation or
tracheostomy). Further, most infants achieved motor milestone and growth
parameter gains generally consistent with normal development, such as
head control, independent sitting, standing and walking independently,
as measured by validated scales.

Three infants experienced AEs considered possibly related to SPINRAZA by
the investigator, all of which were resolved. No infants have
discontinued or withdrawn from the study due to AEs, and no new safety
concerns have been identified.

“The results from NURTURE are significant, as they continue to
demonstrate the importance of beginning SPINRAZA treatment as soon as
possible after an SMA diagnosis and the major impact that early
treatment may have across a broad range of SMA populations,” said
Sandrock.

For more information about SPINRAZA and U.S. prescribing information,
visit www.SPINRAZA.com.

The CHERISH and NURTURE slide presentations will be available
concurrently with the AAN sessions on the Investor section of the Biogen
company website, www.Biogen.com.

SPINRAZA Program Status

Biogen licensed the global rights to develop, manufacture and
commercialize SPINRAZA from Ionis Pharmaceuticals (NASDAQ:IONS), a
leader in antisense therapeutics. Biogen and Ionis conducted an
innovative clinical development program that moved SPINRAZA from its
first dose in humans in 2011 to its first regulatory approval by the
United States Food and Drug Administration (FDA) in 2016.2

SPINRAZA was first approved by the FDA on December 23, 2016 within three
months of regulatory filing for the treatment of SMA in pediatric and
adult patients. In April 2017, the Committee for Medicinal Products for
Human Use (CHMP) of the European Medicines Agency (EMA) adopted a
positive opinion recommending the granting of a marketing authorization
for SPINRAZA for the treatment of 5q SMA, following review under an
Accelerated Assessment program. A decision from the European Commission
(EC) is expected in the next few months. Biogen has also submitted
regulatory filings in Japan, Canada, Australia and Switzerland and plans
to initiate additional filings in other countries in 2017.

About SMA2-6

Spinal muscular atrophy (SMA) is characterized by loss of motor neurons
in the spinal cord and lower brain stem, resulting in severe and
progressive muscular atrophy and weakness. Ultimately, individuals with
the most severe type of SMA can become paralyzed and have difficulty
performing the basic functions of life, like breathing and swallowing.

Due to a loss of, or defect in, the SMN1 gene, people with SMA do
not produce enough survival motor neuron (SMN) protein, which is
critical for the maintenance of motor neurons. The severity of SMA
correlates with the amount of SMN protein. People with Type 1 SMA, the
form that requires the most intensive and supportive care, produce very
little SMN protein and do not achieve the ability to sit without support
or live beyond two years without respiratory support. People with Type 2
or Type 3 SMA produce greater amounts of SMN protein and have less
severe, but still life-altering forms of SMA.

To support awareness and education about SMA, Biogen has launched
Together in SMA in the United States and Japan. Together in SMA is a
program created to provide informational materials and resources to the
SMA community. Learn more at www.TogetherinSMA.com (U.S.-only)
and www.TogetherinSMA.jp/ (Japan-only).

About SPINRAZA™ (nusinersen)

SPINRAZA is being developed globally for the treatment of SMA.

SPINRAZA is an antisense oligonucleotide (ASO), using Ionis
Pharmaceuticals’ proprietary antisense technology, that is designed to
treat SMA caused by mutations or deletions in the SMN1 gene
located in chromosome 5q that leads to SMN protein deficiency. SPINRAZA
alters the splicing of SMN2 pre-mRNA in order to increase
production of full-length SMN protein.7 ASOs are short
synthetic strings of nucleotides designed to selectively bind to target
RNA and regulate gene expression. Through use of this technology,
SPINRAZA has the potential to increase the amount of full-length SMN
protein in patients with SMA.

SPINRAZA is administered via intrathecal injection, which delivers
therapies directly to the cerebrospinal fluid (CSF) around the spinal
cord,8 where motor neurons degenerate in patients with SMA
due to insufficient levels of SMN protein.9

The most common adverse reactions reported for SPINRAZA were lower
respiratory infection, upper respiratory infection and constipation.
Serious adverse reactions of atelectasis were more frequent in
SPINRAZA-treated patients. Coagulation abnormalities and
thrombocytopenia, including acute severe thrombocytopenia, have been
observed after administration of some antisense oligonucleotides.
Individuals may be at increased risk of bleeding complications. Renal
toxicity has been observed after administration of some antisense
oligonucleotides. SPINRAZA is present in and excreted by the kidney.

As part of Biogen’s commitment to patients and families living with SMA,
the company has launched SMA360°™, which provides certain services that
address nonmedical barriers to access in the U.S. These include
logistical assistance, product education, insurance benefits
investigations and financial assistance. A list of the SMA360° offerings
is available at www.SPINRAZA.com.

SMA360° services from Biogen are available only to those eligible
patients who have been prescribed SPINRAZA in the U.S. To learn more
about the program and receive additional information about these
services, please contact an SMA Support Coordinator at
1-844-4SPINRAZA (1-844-477-4672) Monday-Friday 8:30 a.m.-8:00 p.m. EST.

About Biogen

Through cutting-edge science and medicine, Biogen discovers, develops
and delivers innovative therapies worldwide for people living with
serious neurological and neurodegenerative diseases. Founded in 1978,
Biogen is a pioneer in biotechnology and today the Company has the
leading portfolio of medicines to treat multiple sclerosis, has
introduced the first and only approved treatment for spinal muscular
atrophy, and is at the forefront of neurology research for conditions
including Alzheimer’s disease, Parkinson’s disease and amyotrophic
lateral sclerosis. Biogen also manufactures and commercializes
biosimilars of advanced biologics. For more information, please visit www.biogen.com.
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Biogen Safe Harbor

This press release contains forward-looking statements, including
statements relating to the potential benefits, safety and efficacy of
SPINRAZA, the status of current regulatory filings, and plans for
additional regulatory filings in other jurisdictions. These statements
may be identified by words such as “believe,” “except,” “may,” “plan,”
“potential,” “will” and similar expressions, and are based on our
current beliefs and expectations. You should not place undue reliance on
these statements. These statements involve risks and uncertainties that
could cause actual results to differ materially from those reflected in
such statements, including uncertainty of success in commercialization
of SPINRAZA, which may be impacted by, among other things, the level of
preparedness of healthcare providers to treat patients, difficulties in
obtaining or changes in the availability of reimbursement for SPINRAZA,
the effectiveness of sales and marketing efforts, problems with the
manufacturing process for SPINRAZA, the occurrence of adverse safety
events, failure to obtain regulatory approvals in other jurisdictions,
failure to protect intellectual property and other proprietary rights,
product liability claims, third party collaboration risks, and the other
risks and uncertainties that are described in the Risk Factors section
of Biogen’s most recent annual or quarterly report and in other reports
Biogen has filed with the U.S. Securities and Exchange Commission (SEC).
Any forward-looking statements speak only as of the date of this press
release and we assume no obligation to update any forward-looking
statement.